The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 3 of 3 for:    HT-100
Previous Study | Return to List | Next Study

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01847573
Recruitment Status : Terminated (Dosing stopped)
First Posted : May 7, 2013
Last Update Posted : September 3, 2020
Sponsor:
Information provided by (Responsible Party):
Processa Pharmaceuticals

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: HT-100 Phase 1 Phase 2

Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
Study Start Date : May 2013
Actual Primary Completion Date : March 30, 2016
Actual Study Completion Date : March 30, 2016

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Cohort 1: HT-100 tablet, Dose 1
  • Single dose administration: Dose 1
  • Multiple dose administration: Dose 1
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 2: HT-100 tablet, Dose 2
  • Single dose administration: Dose 2
  • Multiple dose administration: Dose 2
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 3: HT-100 tablet, Dose 3
  • Single dose administration: Dose 3
  • Multiple dose administration: Dose 3
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 4a: HT-100 tablet, Dose 4
  • Single dose administration: Dose 4
  • Multiple dose administration: Dose 4
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 4b: HT-100 tablet, Dose 5
* Multiple dose administration: Dose 5
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Experimental: Cohort 5: HT-100 tablet, Dose 6
* Multiple dose administration: Dose 5
 Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys [ Time Frame: 1 week ]
    Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing


Secondary Outcome Measures :
  1. Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys [ Time Frame: 1 week ]
    Halofuginone plasma concentrations

  2. Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks [ Time Frame: 4 weeks ]
    Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing

  3. Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys [ Time Frame: 4 weeks ]

    Pharmacodynamic measures relevant to DMD pathology:

    • Pulmonary function
    • Motor function
    • Muscle composition
    • Biochemical and imaging markers


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   6 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Ambulatory or non-ambulatory
  • Diagnosis of DMD with confirmation of minimal to no dystrophin
  • Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)

Main Exclusion Criteria:

  • Recent, substantial change in use of cardiac medications or medications affecting muscle function
  • Inability to undergo magnetic resonance imaging (MRI)
  • Significantly compromised cardio-respiratory function
  • Prior treatment with another investigational product in past 6 months
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01847573


Locations
Layout table for location information
United States, California
University of California, Davis Medical Center
Sacramento, California, United States, 95817
United States, Maryland
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Processa Pharmaceuticals
Investigators
Layout table for investigator information
Study Director: Diana M Escolar, MD Akashi Therapeutics
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Additional Information:

Layout table for additonal information
Responsible Party: Processa Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01847573    
Other Study ID Numbers: HALO-DMD-01
HALO ( Other Identifier: Akashi Therapeutics )
First Posted: May 7, 2013    Key Record Dates
Last Update Posted: September 3, 2020
Last Verified: March 2019
Keywords provided by Processa Pharmaceuticals:
halofuginone hydrobromide
anti-fibrotic
anti-inflammatory
muscle regeneration
protein synthesis inhibitor
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Halofuginone
Antineoplastic Agents
Coccidiostats
 Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors